The European Medicines Agency’s CHMP backed Minoryx’s ALD therapy while rejecting Zevra’s Meplyffa in a set of rare-disease opinions that highlight uneven European momentum. Minoryx’s neurological drug had faced multiple hurdles, and CHMP recommendations now represent a pivotal step toward potential EU authorization. Zevra, by contrast, received a setback in Europe despite prior U.S. approval, underscoring how efficacy or benefit-risk assessments can diverge between jurisdictions and over time. For Minoryx, CHMP support is a key regulatory milestone likely to reshape investor and partner expectations. For Zevra, the rejection forces reassessment of the European path so close to its U.S. launch. Both outcomes reinforce the importance of Europe-specific evidence packages and post-approval strategy when building global rare disease footprints.